Rare Disease Day 2021 Recap

Observed annually on the last day of February, Rare Disease Day seeks to raise awareness among the general public, as well as policymakers, public authorities, industry representatives, scientists, and health professionals.

 

Due to the COVID-19 pandemic, activities this year were mainly virtual. But that did not stop patients, caregivers, researchers, clinicians, advocates and patient organizations like MDC from sharing stories on social media platforms, participating in academic and community discussions, and holding virtual campaigns to “recognize rare.” In the days leading up to Rare Disease Day, MDC celebrated by bringing awareness to different groups of neuromuscular disorders. MDC also shared powerful stories of on how children and adults are managing their rare disease during the COVID-19 pandemic.

The stories highlighted the resilience of individuals and their families, the benefits of virtual tele-care and programs for those with mobility challenges and the positive lessons the pandemic has offered.

Click here if you missed the stories!

 

Rare Disease Day also served as a great opportunity to share exciting announcements, like:

    • The declaration of International Myotonic Dystrophy Day as September 15, 2021. This day is a collaboration of dozens of myotonic dystrophy groups and organisations from around the globe – each dedicated to helping raise awareness of the condition in their local geographies on September 15, 2021 and beyond. The NMD4C & MDC are proud to be a part of this global alliance in declaring September 15th International Myotonic Dystrophy Awareness Day!

 

 

  • The results of the European Joint Programme for Rare Diseases (EJP RD) JTC 2020 call on pre-clinical research were announced. ProDGNE, a project which will unify European and Canadian (Dr. Hanns Lochmuller) efforts to develop an innovate therapeutic approach for GNE myopathy, an ultra-rare muscle disease, obtained co-funding under the EJP RD JTC 2020 call on pre-clinical research.

 

 

  • MDC has been in partnership with CIHR in the EJP RD competition since 2013. This year, MDC together with CIHR will support the FSHD (EPiTHE4FSHD) (Facioscapulohumeral muscular dystrophy) study (Dr. Rima Al-Awar), which aims to use cellular and animal models of FSHD to investigate a novel pharmacological approach that could represent a promising therapeutic option for patients.

 

Rare Disease Day also helped bring the neuromuscular community up in media. Muscular Dystrophy Canada and Jesse’s Journey were pleased be part of a 2021 Rare Disease Day initiative called Canada’s Rare Voices. This is an online program, the largest of its kind in recent memory, devoted entirely to increasing the visibility of Canada’s rare disorder communities. MDC was featured in a story about the power of partnerships – teaming up to take on neuromuscular disorders.

Please check out the article here!

image001

Read next...

AGAMREE (vamorolone) Approved treatment for DMD in Canada

A Milestone in Neuromuscular Care: AGAMREE® (vamorolone) Approved as the First Treatment for Duchenne Muscular Dystrophy in Canada

Health Canada has approved AGAMREE® (vamorolone), the first-ever treatment for Duchenne Muscular Dystrophy (DMD) in Canada. Patient Support Program opens for enrollment on October 10, 2025.

Website - SBMA published on CMAJ.png

New Best Practice Recommendations for SBMA Care in Canada

Congratulations to Dr. Kerri Schellenberg, the team of Canadian experts, NMD4C Steering Committee members and Investigators, on publishing the first Canadian SBMA care guidelines!

2025 NMD4C ANNUAL MEETING

Advancing Neuromuscular Research and Care: Highlights from the 2025 NMD4C Annual Meeting

On September 11, NMD4C hosted its 2025 Annual Meeting in Ottawa, bringing together clinicians, researchers, trainees, and partners for a full day of collaboration, strategic dialogue, and community-building.

2025 Clinical Summer School

NMD4C Hosts Inaugural Clinical Summer School to Empower Canada’s Future Neuromuscular Leaders

On September 10–11, NMD4C hosted its inaugural Clinical Summer School in Ottawa, bringing together 19 neuromuscular clinical fellows from across Canada for two days of hands-on training, mentorship, and networking. Led by national experts and co-chaired by Drs. Gordon Jewitt and Marianne Nury, the program complemented NMD4C’s accredited lecture series and fostered peer connection through dedicated community-building sessions.

2026 Neuromuscular Postdoc Research Fellowship Competition

2026 Neuromuscular Postdoctoral Research Fellowships Funding Competition is Now Open!

Apply Or Share this opportunity with the trainees in your network — Deadline: October 14, 2025. Hosted by Neuromuscular Disease Network for Canada (NMD4C) and Muscular Dystrophy Canada (MDC)

Clinical Fellowship Awardees

Announcing the 2026 Neuromuscular Clinical Fellowship Award Recipients

We are excited to share that the 2026 Neuromuscular Clinical Fellowship Awards have been awarded! These fellowships strengthen clinical training in neuromuscular medicine and help prepare the next generation of specialists who will advance neuromuscular care across Canada.